Laura A Adang, M.D., PhD

faculty photo
Assistant Professor of Neurology at the Children's Hospital of Philadelphia
Department: Neurology

Contact information
The Children's Hospital of Philadelphia
3400 Civic Blvd.
Philadelphia, PA 19104
Office: 215-590-1710
Education:
BS (Interdisciplinary Studies: Neurology - Magna cum laude with Honors)
University of Georgia, 2001.
PhD (Microbiology)
University of Virginia, 2007.
MD
University of Virginia, 2009.
MS (Translational Research )
Penn, 2018.
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Description of Clinical Expertise

Leukodystrophies

Description of Research Expertise

Clinical and biologic outcome measures in the leukodystrophies

Selected Publications

Dominguez Gonzalez CA, Spinner NB, Ahrens-Nicklas RC, Young LR, Voss LA, Reichert SL, Gallo DJ, Cohen JS, Bonkowsky JL, Keller SR, Bennett ML, Pizzino AM, Swantkowski M, Arnold K, Fraser JL, Emerson FJ, Miettunen K, Fatemi A, Van Haren KP, Adang L, Waldman A, Emrick L, Eichler F, Vanderver A.: Reporting ABCD1 Variants as Actionable Secondary Findings on Exome and Genome Sequencing. Genet Med 5(101425), April 2025.

Gavazzi F, Charsar B, Hamilton E, Erler JA, Patel V, Woidill S, Sevagamoorthy A, Helman G, Schmidt J, Pizzino A, Muirhead K, Takanohashi A, Bonkowsky JL, Meyerhoffer K, Simons C, Doi H, Satoko M, Matsumoto N, Delgado MR, Sanchez-Castillo M, Wang J, de Carvalho DR, Tournev I, Chamova T, Jordanova A, Clegg NJ, Nicita F, Bertini E, Teng M, Williams D, Tonduti D, Houlden H, Stellingwerff M, Wassmer E, Garcia-Cazorla A, Bernard G, Mirchi A, Toutounchi H, Wolf NI, van der Knaap MS, Shults J, Adang LA, Vanderver AL.: The natural history of variable subtypes in pediatric-onset TUBB4A-related leukodystrophy. Mol Genet Metab 144(3), March 2025.

Kotes E, Gavazzi F, Woidill S, Sevagamoorthy A, Yang E, Smith V, Dubbs H, Pierce SR, Pucci K, Vithayathil J, Thakur N, Adang LA: Determination of Health Concepts in β-Propeller Protein-Associated Neurodegeneration. Journal of Child Neurology 40(1): 15-25, January 2025.

Mutua S, Sevagamoorthy A, Woidill S, Orchard PJ, Gavazzi F, MacFarland SP, Russo P, Vanderver A, Adang LA.: Characterization of gallbladder disease in metachromatic leukodystrophy across the lifespan. Mol Genet Metab 144(1), January 2025.

Tricoli L, Sase S, Hacker JL, Pham V, Chappell M, Breda L, Hurwitz SN, Tanaka N, Castruccio Castracani C, Guerra A, Hou Z, Schlotawa L, Radhakrishnan K, Hogenauer M, Roche A, Everett J, Bushman F, Kurre P, Ahrens-Nicklas R, Adang LA, Vanderver AL, Rivella S.: Effective gene therapy for metachromatic leukodystrophy achieved with minimal lentiviral genomic integrations. Mol Ther Nucleic Acids 25(36), January 2025.

Pillai NR, Liu N, Li X, Li X, Ahrens-Nicklas R, Adang L, Eisengart JB, Bronken G, Gupta A, Lund TC, Whitley CB, Elsea SH, Orchard PJ.: Bone marrow transplantation reverses metabolic alterations in multiple sulfatase deficiency: a case series. Commun Med 5(1), January 2025.

Porcari GS, Collyer JW, Adang LA, Rajan DS.: Current Advances and Challenges in Gene Therapies for Neurologic Disorders: A Review for the Clinician. Neurol Genet 13(11), January 2025.

Pham V, Tricoli L, Hong X, Wongkittichote P, Castruccio Castracani C, Guerra A, Schlotawa L, Adang LA, Kuhs A, Cassidy MM, Kane O, Tsai E, Presa M, Lutz C, Rivella SB, Ahrens-Nicklas RC: Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiency. Mol Therapy 32(11): 3829-3846, November 2024.

Kornbluh AB, Baldwin A, Fatemi A, Vanderver A, Adang LA, Van Haren K, Sampson J, Eichler FS, Sadjadi R, Engelen M, Orthmann-Murphy JL.: Practical Approach to Longitudinal Neurologic Care of Adults With X-Linked Adrenoleukodystrophy and Adrenomyeloneuropathy. Neurol Genet 10(5): e200192, October 2024.

Peixoto de Barcelos I, Jan AK, Modesti N, Woidill S, Gavazzi F, Isaacs D, D'Aiello R, Sevagamoorthy A, Charlton L, Pizzino A, Schmidt J, van Haren K, Keller S, Eichler F, Emrick LT, Fraser JL, Shults J, Vanderver A, Adang LA.: Systemic complications of Aicardi Goutières syndrome using real-world data. Mol Genet Metab 143(1-2), September-October 2024.

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Last updated: 05/09/2025
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